EUCTR2016-001478-14-IT进行中（未招募）1 期
A PROSPECTIVE, PHASE 3, OPEN LABEL, INTERNATIONALMULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXISWITH rVWF IN SEVERE VON WILLEBRAND DISEASE - rVWF IN PROPHYLAXIS
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Subjects who meet ALL of the following criteria are eligible for this study:
- •1. Subject has a documented diagnosis of severe VWD (baseline VWF:RCo <20 IU/dL) with a history
- •of requiring substitution therapy with von Willebrand factor concentrate to control bleeding:
- •a. Type 1 (VWF:RCo <20 IU/dL) or,
- •b. Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype), Type 2M or,
- •c. Type 3 (VWF:Ag =3 IU/dL).
- •2. Diagnosis is confirmed by genetic testing and multimer analysis, documented in patient history or at
- •3. Subject currently receiving on-demand treatment for whom prophylactic treatment is recommended
- •according to standard of care at the center.
- •4. Has =3 documented spontaneous bleeds requiring VWF treatment during the past 12 months
- •5. Availability of records to reliably evaluate type, frequency and treatment of bleeding episodes
- •during 12 months of on-demand treatment prior to enrollment.
- •6. Subject is =18 years old at the time of screening and has a body mass index =15 but <40 kg/m2.
- •7. If female of childbearing potential, subject presents with a negative blood/urine pregnancy test at
- •screening and agrees to employ adequate birth control measures for the duration of the study.
- •8. Subject is willing and able to comply with the requirements of the protocol.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 14
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •Subjects who meet ANY of the following criteria are not eligible for this study:
- •1. The subject has been diagnosed with Type 2N VWD, pseudo VWD, or another hereditary or
- •acquired coagulation disorder other than VWD (e.g., qualitative and quantitative platelet disorders
- •or elevated prothrombin time (PT)/international normalized ratio [INR] ?1.4).
- •2. The subject has received prophylaxis treatment in the 12 months prior to screening (including those
- •who received treatment once a month for menorrhagia but were not treated for any other bleeds).
- •3. The subject is currently receiving prophylaxis treatment.
- •4. The subject has a history or presence of a VWF inhibitor at screening.
- •5. The subject has a history or presence of a FVIII inhibitor with a titer =0.4 BU (by Nijmegen
- •modified Bethesda assay) or =0.6 BU (by Bethesda assay).
- •6. The subject has a known hypersensitivity to any of the components of the study drugs, such as to
- •mouse or hamster proteins.
- •7. The subject has a medical history of immunological disorders, excluding seasonal allergic
- •rhinitis/conjunctivitis, mild asthma, food allergies or animal allergies.
- •8. The subject has a medical history of a thromboembolic event.
- •9. The subject is HIV positive with an absolute Helper T cell (CD4) count ?200/mm3.
- •10. The subject has been diagnosed with significant liver disease as evidenced by any of the following:
- •serum alanine aminotransferase (ALT) 5 times the upper limit of normal; hypoalbuminemia; portal
- •vein hypertension (e.g., presence of otherwise unexplained splenomegaly, history of esophageal
- •11. The subject has been diagnosed with renal disease, with a serum creatinine level =2.5 mg/dL.
- •12. The subject has a platelet count <100,000/mL at screening.
- •13. The subject has been treated with an immunomodulatory drug, excluding topical treatment (e.g.,
- •ointments, nasal sprays), within 30 days prior to signing the informed consent.
- •14. The subject is pregnant or lactating at the time of enrollment.
- •15. Patient has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection,
- •dysplasia).
- •16. The subject has participated in another clinical study involving another investigational product (IP)
- •or investigational device within 30 days prior to enrollment or is scheduled to participate in another
- •clinical study involving an IP or investigational device during the course of this study.
- •17. The subject has a progressive fatal disease and/or life expectancy of less than 15 months.
- •18. The subject is identified by the investigator as being unable or unwilling to cooperate with study
- •procedures.
- •19. The subject has a mental condition rendering him/her unable to understand the nature, scope and
- •possible consequences of the study and/or evidence of an uncooperative attitude.
- •20. The subject is in prison or compulsory detention by regulatory and/or juridical order
- •21. The subject is member of the study team or in a dependent relationship with one of the study team
- •members which includes close relatives (i.e., children, partner/spouse, siblings and parents) as well
- •as employees.
- •Delay criteria
- •1. If the subject presents with an acute bleeding episodes or acute illness (e.g., influenza, flu-like
- •syndrome, allergic rhinitis/conjunctivitis, non-seasonal asthma) the screening visit will be postponed
- •until the subject has recovered.
研究者
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